Kathryn Hollenbach, Ph. D.
University of California, San Diego

High-dose chemotherapy with bone marrow or stem cell transplant is a treatment that has been increasingly recommended to women with advanced (metastatic) or high-risk primary breast cancer. Studies have shown that this therapy, which uses very high doses of chemotherapy, may lead to better results in women with high-risk disease. The technique of stem cell transplant has been developed over the past decade to allow the use of extremely high doses of chemotherapy. Stem cells are collected, frozen and then used to rescue blood counts following high-dose chemotherapy. However, a major barrier to more widespread use of this treatment is the very high associated costs, typically ranging from $100,000 to $150,000. Because of this high cost, there has been a great deal of resistance on the part of insurers to include this procedure as a covered benefit. Controversy surrounding this has manifested itself by lawsuits, arbitration hearings, testimony before Congress, and attempts at legislation in many states. It is clear that no matter how successful this therapy is, it will have little impact for the majority of women who might benefit from it unless it is more widely available. More widespread availability, in turn, depends on reduction of total costs. One major source of the high cost has been the traditional need for prolonged inpatient hospitalization. However, in the last few years it has become possible to deliver this therapy in a less costly setting. At Scripps Clinic, early discharge programs have been developed which safely and effectively reduce the total duration of hospitalization as well as the total costs associated with this procedure. We have treated over 150 patients with high-dose chemotherapy and stem cell transplants in an outpatient clinic. Preliminary results demonstrate that this program is extremely safe (procedure-related death rate of less than 1.5 %) and highly acceptable to patients. Preliminary financial analysis suggests a 35% to 43 % reduction in total charges for patients treated in the outpatient setting. However, there is no information on the financial impact of this type of program on the patients and their families. Further, effects on patients emotional and psychological health have not been adequately examined.

We propose to conduct a detailed analysis of the financial impact of outpatient transplantation for breast cancer by comparing the total hospital and clinic costs associated with stem cell transplantation among patients receiving traditional inpatient care and those receiving the identical treatment in this innovative model of outpatient care. In addition, we will examine the financial impact on the patients and their families in the two settings to determine whether any shift in costs from insurers to patients and families is occurring. We will also compare the "quality of life" and psychological impact of treatment for the different treatment settings. If outpatient transplants are shown to reduce total costs without unduly burdening patients or their families, substantial barriers to more widespread use of this potentially life-saving therapy should be reduced.

High-dose chemotherapy with bone marrow or stem cell transplant is a treatment option that has been recommended for women with advanced (metastatic) or high risk breast cancer. Stem cell transplant allows the use of extremely high doses of chemotherapy. Stem cells are collected, frozen and then used to rescue blood counts following high-dose chemotherapy. Unfortunately, costs associated with this type of treatment are very high which has limited the availability of this option for many women. One major source of cost has been traditional prolonged inpatient hospitalization. This study compared high-dose chemotherapy with stem cell replacement among breast cancer patients receiving treatment as inpatients (traditional) and as outpatients. Comparison between inpatient and outpatient treated women at Scripps Clinic Blood and Marrow Transplantation Program were compared for cost of treatment, psychological well being of the patient, psychological well being of the caregiver, toxicity associated with treatment and hospital readmissions.

During the study, 57 women received high-dose chemotherapy with stem cell replacement. Forty-eight (84%) agreed to participate in this study. The primary reason for not receiving treatment in the outpatient setting was unavailability of an identified caregiver. Of women who agreed to participate, 31% received treatment as inpatients and 69% received treatment as outpatients. There were no statistically significant differences between inpatient and outpatient treated groups on time to graduation (days from first date of high-dose chemotherapy to date returned to referring oncologist), toxicities, number of hospital readmissions or length of hospital readmission.

Women treated as inpatients accrued mean costs that were significantly higher than those treated as outpatients (p<0.001; adjusted for age and symptom severity). On average outpatients costs were 71% less than inpatient costs. Quality of well being (QWB) was assessed for patients and caregivers before, during and after treatment completion. No differences in QWB were seen between inpatient and outpatient patients or caregivers at baseline or 60 days after treatment. Significant differences in patient QWB scores were observed during treatment with outpatients demonstrating poorer QWB scores (p< 0.001; adjusted for age, symptom severity and baseline QWB). Similar differences were not seen for caregivers. Although outpatients had poorer QWB scores, there were no differences between groups on time to graduation and the negative effects were not observed at follow-up.

Results demonstrate that this therapy, administered in an outpatient setting was associated with significant cost savings and no permanent adverse effects. The significant cost savings support widespread availability of this therapy.